RNA Therapeutics: Antisense Oligonucleotides, siRNA, and mRNA-Based Medicines

The clinical success of mRNA vaccines, siRNA therapeutics, and antisense oligonucleotides has established RNA-based medicines as a new pillar of molecular pharmacology, and this chapter provides a comprehensive review of the mechanistic principles, delivery innovations, and clinical landscape of this therapeutic class. It covers the molecular biology of RNA interference, the design and optimisation of therapeutic oligonucleotides, lipid nanoparticle and conjugate delivery systems, and the regulatory pathway for RNA-based drugs. Approved therapies and the pipeline of RNA medicines in clinical development are reviewed across disease areas.